Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Sep 27, 2021

Keywords

ClinConnect Summary

The ARTEGENE trial is studying a new gene therapy for children and young adults with a specific kind of severe combined immunodeficiency (SCID) caused by a mutation in the DCLRE1C gene, also known as the Artemis gene. This condition makes it difficult for the body to fight infections. The trial aims to see if transplanting a single dose of the patient’s own special stem cells, which have been modified to include a healthy copy of the DCLRE1C gene, is safe and effective in helping these patients.

To be eligible for the study, participants must be 47 months old or younger and have a confirmed diagnosis of SCID due to mutations in the Artemis gene. They also need to not have an immediate compatible donor for a bone marrow transplant. If enrolled, participants will receive the gene therapy and will need to return for follow-up visits over the next two years to monitor their health and the therapy's effects. This is an important opportunity for families facing this challenging condition, as it may offer a new treatment option that could improve their child’s immune function and overall health.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patient to 47 months
• • SCID patients with confirmed biallelic mutations in the Artemis (DCLRE1C) gene even in the case of leaky forms characterised by a residual activity
• • Absence of an HLA genoidentical donor or without rapidly available HLA-compatible unrelated donor (within six weeks of diagnosis)
• • The patient can be treated by gene therapy without delay in case of active life threatening infections compromising the short-term prognosis and for which the delay in finding a phenoidentical donor is incompatible with the patient's condition of health. Active life threatening infections are defined as: viral respiratory infection, CMV infection, adenovirus infection, disseminated BCGitis or other infections grade ≥ 4 according to CTCAE scale
• • Beneficiary of a social security scheme
• • Parental, guardian's patient signed informed consent.
• • Exclusion Criteria
• • Unwillingness to return for follow-up during the first 2 years study and the long term follow-up
• • HIV-1 or 2 or HTLV1 infections
• • Hypersensitivity to G-CSF, busulfan or Fludarabine
• • Unable to tolerate general anesthesia and/or marrow harvest or peripheral blood stem cell collection (apheresis) or insertion of central venous catheter.